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精准制导治疗高危儿科癌症。

Precision-guided treatment in high-risk pediatric cancers.

机构信息

Children's Cancer Institute, Lowy Cancer Research Centre, UNSW Sydney, Sydney, New South Wales, Australia.

School of Clinical Medicine, UNSW Medicine & Health, UNSW Sydney, Sydney, New South Wales, Australia.

出版信息

Nat Med. 2024 Jul;30(7):1913-1922. doi: 10.1038/s41591-024-03044-0. Epub 2024 Jun 6.

DOI:10.1038/s41591-024-03044-0
PMID:38844796
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11271405/
Abstract

Recent research showed that precision medicine can identify new treatment strategies for patients with childhood cancers. However, it is unclear which patients will benefit most from precision-guided treatment (PGT). Here we report consecutive data from 384 patients with high-risk pediatric cancer (with an expected cure rate of less than 30%) who had at least 18 months of follow-up on the ZERO Childhood Cancer Precision Medicine Program PRecISion Medicine for Children with Cancer (PRISM) trial. A total of 256 (67%) patients received PGT recommendations and 110 (29%) received a recommended treatment. PGT resulted in a 36% objective response rate and improved 2-year progression-free survival compared with standard of care (26% versus 12%; P = 0.049) or targeted agents not guided by molecular findings (26% versus 5.2%; P = 0.003). PGT based on tier 1 evidence, PGT targeting fusions or commenced before disease progression had the greatest clinical benefit. Our data show that PGT informed by comprehensive molecular profiling significantly improves outcomes for children with high-risk cancers. ClinicalTrials.gov registration: NCT03336931.

摘要

最近的研究表明，精准医学可以为儿童癌症患者确定新的治疗策略。然而，目前尚不清楚哪些患者将从精准指导治疗（PGT）中获益最大。在这里，我们报告了连续 384 名患有高危儿科癌症（预期治愈率低于 30%）患者的随访时间至少为 18 个月的结果，这些患者均参加了 ZERO 儿童癌症精准医学计划 PRISM 试验。共有 256 名（67%）患者接受了 PGT 推荐，110 名（29%）患者接受了推荐的治疗。PGT 导致客观缓解率提高了 36%，与标准治疗（26%比 12%；P=0.049）或未根据分子发现指导的靶向药物治疗（26%比 5.2%；P=0.003）相比，2 年无进展生存率得到了改善。基于 1 级证据的 PGT、针对融合的 PGT 或在疾病进展前开始的 PGT 具有最大的临床获益。我们的数据表明，基于全面分子分析的 PGT 显著改善了高危癌症患儿的预后。临床试验注册：NCT03336931。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/43f1/11271405/54e3ae96913e/41591_2024_3044_Fig11_ESM.jpg
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https://cdn.ncbi.nlm.nih.gov/pmc/blobs/43f1/11271405/341cd550e8bf/41591_2024_3044_Fig4_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/43f1/11271405/fa2b8bf5ea6a/41591_2024_3044_Fig5_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/43f1/11271405/7e5249a9adbc/41591_2024_3044_Fig6_ESM.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/43f1/11271405/d46a7ce4077e/41591_2024_3044_Fig7_ESM.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/43f1/11271405/9c57d5eded6d/41591_2024_3044_Fig8_ESM.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/43f1/11271405/2cfc8e6d9a06/41591_2024_3044_Fig9_ESM.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/43f1/11271405/6e0b727f60ae/41591_2024_3044_Fig10_ESM.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/43f1/11271405/54e3ae96913e/41591_2024_3044_Fig11_ESM.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/43f1/11271405/b52cf48feeec/41591_2024_3044_Fig1_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/43f1/11271405/1f3415008e29/41591_2024_3044_Fig2_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/43f1/11271405/9a3b0808bb2a/41591_2024_3044_Fig3_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/43f1/11271405/341cd550e8bf/41591_2024_3044_Fig4_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/43f1/11271405/fa2b8bf5ea6a/41591_2024_3044_Fig5_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/43f1/11271405/7e5249a9adbc/41591_2024_3044_Fig6_ESM.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/43f1/11271405/d46a7ce4077e/41591_2024_3044_Fig7_ESM.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/43f1/11271405/9c57d5eded6d/41591_2024_3044_Fig8_ESM.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/43f1/11271405/2cfc8e6d9a06/41591_2024_3044_Fig9_ESM.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/43f1/11271405/6e0b727f60ae/41591_2024_3044_Fig10_ESM.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/43f1/11271405/54e3ae96913e/41591_2024_3044_Fig11_ESM.jpg

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本文引用的文献

[1]
Precision Medicine for Childhood Cancer: Current Limitations and Future Perspectives.

JCO Precis Oncol. 2024-1

[2]
Molecular diagnostics enables detection of actionable targets: the Pediatric Targeted Therapy 2.0 registry.

Eur J Cancer. 2023-2

[3]
A Review of Patient-Reported Outcome Measures in Childhood Cancer.

Children (Basel). 2022-9-30

[4]
Molecular profiling identifies targeted therapy opportunities in pediatric solid cancer.

Nat Med. 2022-8

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The European MAPPYACTS Trial: Precision Medicine Program in Pediatric and Adolescent Patients with Recurrent Malignancies.

Cancer Discov. 2022-5-2

[6]
In vitro and in vivo drug screens of tumor cells identify novel therapies for high-risk child cancer.

EMBO Mol Med. 2022-4-7

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Cancer Discov. 2021-11

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Cancer Discov. 2021-12-1

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Whole genome, transcriptome and methylome profiling enhances actionable target discovery in high-risk pediatric cancer.

Nat Med. 2020-11

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Long-term overall survival and prognostic score predicting survival: the IMPACT study in precision medicine.

J Hematol Oncol. 2019-12-30

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